Loading patent…
OwnerPascal Neuville, Sebatien Ribault +2inventors
InventorsPascal Neuville, Sebatien Ribault, Valerie Calenda, Melanie Frauli
Estimating the term…
The present invention concerns chimeric constructs comprising a skeletal alpha-actin gene promoter operably linked with at least a skeletal muscle-specific enhancer of a human gene. It also provides an expression cassette comprising such a chimeric construct to control expression of a gene of interest. The invention also relates to a vector, a viral particle, an eukaryotic host cell, a pharmaceutical composition comprising said expression cassette and their use for specific expression in skeletal muscle cells as well as for therapeutic or prophylactic purposes. Finally, the present invention also provides the therapeutic use of an expression cassette, a vector and a viral particle comprising a gene of interest placed under the control of a skeletal alpha-actin gene promoter and a muscle-specific enhancer, especially for treating peripheral ischemia.
A chimeric construct for the expression of a gene of interest in a host cell or organism comprising at least (i) a skeletal alpha-actin gene promoter operably linked with at least (ii) a skeletal muscle-specific enhancer of a human gene.
The chimeric construct according to any of the preceeding claims, wherein said skeletal muscle-specific enhancer is selected from the group consisting of enhancers obtained from the human creatine kinase gene, the human beta-enolase (ENO-3) gene, the human myogenin gene and the human troponin gene.
An expression cassette comprising a gene of interest placed under the control of a chimeric construct according to any one of the preceeding claims, allowing its expression in a host cell or organism.
Use of an expression cassette, a vector or a viral particle comprising a gene of interest placed under the control of at least (i) a skeletal alpha-actin gene promoter and (ii) a muscle-specific enhancer for the preparation of a drug for the treatment or the prevention of a cardiovascular disease in a human or animal organism by gene therapy.
Dependencies are inferred from the claim text.